No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
8 – 25
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
20 (estimated)
Sponsor
Stephan Grupp MD PhD · Other
Who this trial is looking for
This trial is looking for children and young adults with severe autoimmune diseases like systemic lupus erythematosus or systemic sclerosis. Participants will receive high-dose therapy followed by a stem cell transplant to help reset their immune system.
Are You a Good Fit for This Trial?
You may be able to join if
I am between 8 and 25 years old
I have severe systemic lupus erythematosus or systemic sclerosis
I have good organ function
I do not have active infections
You may not be able to join if
I have had a stem cell or solid organ transplant before
I am pregnant
I am participating in another clinical trial for a new drug
I have serious health issues that would make treatment unsafe
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
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A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy ha…
A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Age 8 ≤ 25 years at time of enrollment.
2. Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
3. Adequate organ function status
4. No active, untreated infections.
Exclusion Criteria:
1. Previous hematopoietic stem cell transplant (HSCT) or…
Inclusion Criteria:
1. Age 8 ≤ 25 years at time of enrollment.
2. Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
3. Adequate organ function status
4. No active, untreated infections.
Exclusion Criteria:
1. Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant
2. Pregnancy
3. Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
4. Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy
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